Catabasis Pharmaceuticals Presents Preclinical Data Showing Potential for Bone Preservation with Edasalonexent in Duchenne Mu...
June 21 2019 - 9:45AM
Business Wire
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today presented new preclinical data
showing preserved bone health with edasalonexent in contrast to
negative effects of the corticosteroid prednisolone in a mouse
model of Duchenne muscular dystrophy (DMD). Edasalonexent is a
novel NF-kB inhibitor in Phase 3 development for the treatment of
DMD. The data were presented at the Symposium on Muscle-Bone
Interaction in Duchenne Muscular Dystrophy.
Bone health is important to those affected by Duchenne as many
patients experience long bone and/or vertebral fractures before the
age of 13. In the preclinical study sponsored by Catabasis,
prednisolone treatment negatively impacted bone health in mdx mice
(mouse model of DMD) compared to control, whereas edasalonexent
treatment showed bone sparing effects compared to the bone loss
seen with prednisolone. Mice treated with prednisolone had
significantly weaker bones (both cortical density and cortical
thickness) and also grew less as assessed by femur length compared
to control mdx mice. These results were seen following 6 months of
treatment of clinically relevant doses of prednisolone or
edasalonexent. The mice receiving 6 months of edasalonexent had
preserved cortical density, cortical thickness and femur length,
similar to the control mice.
Corticosteroids, such as prednisolone, can negatively impact
bone health by increasing osteoclast apoptosis, reducing
ossification and leading to increased bone resorption and
osteoporosis. Catabasis believes that the treatment of DMD by
inhibiting NF-kB with edasalonexent has the potential to reduce
bone loss and enhance new bone growth in those affected by DMD by
increasing osteoblast maturation and decreasing osteoclast
differentiation and function.
Boys with DMD typically have decreased height compared to boys
that do not have DMD and the adverse effects of corticosteroids
include decreased vertical growth. In the Phase 2 MoveDMD® trial
and open-label extension, over a 72-week period, boys receiving
edasalonexent grew taller by an average of 2.1 inches per year,
increases that were consistent with the growth curves of unaffected
boys. The effects of edasalonexent on bone health are being further
studied in DMD in the ongoing global Phase 3 PolarisDMD trial,
which includes dual-energy X-ray absorptiometry (DXA) to assess
bone density and body composition and standardized lumbosacral
spine films to assess for fractures at baseline and following 1
year of treatment.
The Phase 3 PolarisDMD trial is a one-year, randomized,
double-blind, placebo-controlled trial evaluating the efficacy and
safety of edasalonexent in patients with DMD. Catabasis plans to
enroll approximately 125 patients ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at
least 6 months. Boys on a stable dose of eteplirsen may be eligible
to enroll. The primary efficacy endpoint is change in the North
Star Ambulatory Assessment score after 12 months of treatment with
edasalonexent compared to placebo. Key secondary endpoints include
the age-appropriate timed function tests: time to stand, 4-stair
climb and 10-meter walk/run. Assessments of growth, cardiac and
bone health are also included as important potential areas of
differentiation. Two boys are receiving 100 mg/kg/day of
edasalonexent for each boy that receives placebo, and, after 12
months, all boys are expected to receive edasalonexent in the
open-label extension study GalaxyDMD. Top-line results from the
Phase 3 PolarisDMD trial are expected in the second half of
2020.
About Edasalonexent (CAT-1004) Edasalonexent (CAT-1004)
is an investigational oral small molecule that is being developed
as a potential therapy for all patients affected by DMD, regardless
of their underlying mutation. Edasalonexent inhibits NF-kB, which
is a key link between loss of dystrophin and disease progression in
DMD. NF-kB has a fundamental role in skeletal and cardiac muscle
disease in DMD. We are currently enrolling our global Phase 3
PolarisDMD trial to evaluate the efficacy and safety of
edasalonexent for registration purposes. Edasalonexent is also
being dosed in the open-label extension trial GalaxyDMD. In our
MoveDMD Phase 2 trial and open-label extension, we observed that
edasalonexent preserved muscle function and substantially slowed
disease progression compared to rates of change in a control
period, and significantly improved biomarkers of muscle health and
inflammation. The FDA has granted orphan drug, fast track, and rare
pediatric disease designations and the European Commission has
granted orphan medicinal product designation to edasalonexent for
the treatment of DMD. For a summary of clinical results, please
visit www.catabasis.com.
About Catabasis At Catabasis Pharmaceuticals, our mission
is to bring hope and life-changing therapies to patients and their
families. Our lead program is edasalonexent, an NF-kB inhibitor in
development for the treatment of Duchenne muscular dystrophy. Our
global Phase 3 PolarisDMD trial is currently enrolling boys
affected by Duchenne. For more information on edasalonexent and our
Phase 3 PolarisDMD trial, please visit www.catabasis.com.
Forward Looking Statements Any statements in this press
release about future expectations, plans and prospects for the
Company, including statements about future clinical trial plans
including, among other things, statements about the Company’s
global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and
safety of edasalonexent for registration purposes, including the
anticipated timing for completion of enrollment and top-line
results, the effect of edasalonexent on the preservation of bone in
those affected by Duchenne muscular dystrophy, and other statements
containing the words “believes,” “anticipates,” “plans,” “expects,”
“may” and similar expressions, constitute forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including: uncertainties inherent in the
initiation and completion of preclinical studies and clinical
trials and clinical development of the Company’s product
candidates; whether interim results from a preclinical or clinical
trial will be predictive of the final results of the trial or the
results of future trials; expectations for regulatory approvals to
conduct trials or to market products; availability of funding
sufficient for the Company’s foreseeable and unforeseeable
operating expenses and capital expenditure requirements; other
matters that could affect the availability or commercial potential
of the Company’s product candidates; and general economic and
market conditions and other factors discussed in the “Risk Factors”
section of the Company’s Quarterly Report on Form 10-Q for the year
ended March 31, 2019, which is on file with the Securities and
Exchange Commission, and in other filings that the Company may make
with the Securities and Exchange Commission in the future. In
addition, the forward-looking statements included in this press
release represent the Company’s views as of the date of this press
release. The Company anticipates that subsequent events and
developments will cause the Company’s views to change. However,
while the Company may elect to update these forward-looking
statements at some point in the future, the Company specifically
disclaims any obligation to do so. These forward-looking statements
should not be relied upon as representing the Company’s views as of
any date subsequent to the date of this release.
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Investor and Media Contact Andrea
Matthews Catabasis Pharmaceuticals, Inc. T: (617) 349-1971
amatthews@catabasis.com
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